On Wednesday 28th February 2018 a joint patient and clinician event was held at the Queen Elizabeth Hospital, Birmingham to celebrate RaDaR’s achievements to date and consider where we go in the next ten years.

The Agenda, a report from the day and pdfs of the presentation can be found below.

At the meeting, Dicerna announced the expansion of its drug development programs to encompass all forms of primary hyperoxaluria (PH). Based on new pre-clinical data presented in Tenerife, the investigational therapy DCR-PHXC has the potential to treat all forms of PH.

The webinar will provide an overview of the following research topics:

• The need for new treatment strategies for all types of PH
• The role of the LDHA gene in the pathogenesis of PH
• The DCR-PHXC preclinical data demonstrate potent, durable, and precise knockdown of LDHA in animal models of PH1, PH2 and idiopathic PH, and show a simple, direct linear relationship between LDHA inhibition and oxalate production
• Clinical development plans and timelines for DCR-PHXC

The Summer issue of the RaDaR Newsletter features updates from the IgA Nephropathy and aHUS groups, details of a Cystinosis patient information event in Birmingham and important information about the forthcoming Ethics amendment.

Due to unforeseen circumstances the National Cystinosis Study Day has had to be postponed. The event will hopefully be re-scheduled for Spring 2018. Details will be published here once they are confirmed.

A National Cystinosis Study Day will be held on Tuesday 19th September 2017 from 9.30am – 4.30pm at the Post Graduate Centre, Queen Elizabeth Hospital, Birmingham.

Patients and Health Professionals with an interest in Cystinosis are welcome.